2018 BIOPHARMACEUTICAL RESEARCH INDUSTRY PROFILE AND TOOLKIT

INDUSTRY PROFILE

At biopharmaceutical companies across America, people go to work every day with the mission of helping patients. We stand for hope, treatments and cures that make a difference in millions of patients’ lives.

We are committed to ensuring access for all patients to the medicines they need and for preserving a strong economy with vibrant research and valuable jobs. We also stand behind the medicines we create and the value they provide not only to patients, but across the health care system.

We Drive Innovation

We are in a new era of medicine where breakthrough science and personalized therapies are transforming the way we treat patients. We have invested half a trillion dollars over the last decade and these investments are just beginning to pay off. The progress we're seeing today revolutionizing the way we treat disease. There are about 7,000 medicines in development globally impacting U.S. patients. Across the drug development pipeline, 74 percent have the potential to be first‑in‑class treatments.

Innovative medicines keep patients healthy and out of the hospital, reducing the need for costly medical care and more.

Medicines are our best bet for tackling the biggest cost driver‑chronic disease‑which account for 90 percent of health care spending.

We Support a Strong Economy

Investments in biopharmaceutical innovation are driving contributions to the American economy and solidifying America’s role as a leader in medical innovation. In 2016, biopharmaceutical companies invested about $90 billion in R&D in the U.S. The industry also supports 4.7 million jobs across the country, and over 800,000 employees go to work every day to create new treatments and cures for patients.

We Support Patient Access to Care

Changes in insurance design, including the growing use of deductibles and coinsurance for prescription medicines, create affordability challenges for many patients. Patients enrolled in high deductible plans may pay out of pocket before prescriptions are covered, while patients with coinsurance are responsible for up to 40 percent.

Research shows high cost sharing is associated with lower medication adherence and increased abandonment rates.

We Believe in Our Market-Based System

New medicines are transforming care for patients fighting debilitating diseases. Yet the share of total health spending devoted to prescription medicines remains constant at 14.1 percent.

This is possible because the market-based system in the United States promotes incentives for innovation while leveraging competition to control costs. The U.S. market is highly competitive, with negotiation by payers and competition from brand and generic alternatives. Ninety percent of prescriptions filled by patients are generics that cost a fraction of the price of the brand medicine.

One reason the current marketplace for medicines has been successful in controlling costs is that health insurers and pharmacy benefit managers (PBMs) are powerful, sophisticated purchasers who use their leverage to negotiate discounts and rebates off the “list prices” of medicines. In fact, these discounts and rebates have grown in recent years.

While continued growth in rebates and discounts has kept price growth for brand medicines growing at the slowest rate in years, it doesn’t feel that way for many patients. That is because too often, negotiated savings are not always shared with patients who are increasingly being asked to pay more out‑of‑pocket for innovative medicines.

ENSURING AFFORDABILITY FOR PATIENTS

We need to ensure that health insurance works the way it is intended and offers meaningful protection from out-of-pocket costs for the sickest of patients. To improve patient access and affordability, insurers and PBMs should share more of the discounts and rebates they negotiate with biopharmaceutical companies directly with patients at the point of sale. In addition, insurers and PBMs should have to count all cost sharing patients are asked to pay toward the deductible and out-of-pocket maximum, instead of ignoring amounts paid with the help of cost sharing assistance provided by biopharmaceutical companies.

PROMOTING VALUE-DRIVEN HEALTH CARE

In order for biopharmaceutical companies to become partners—rather than simply vendors—we need to modernize regulations developed for a fee-for-service era. Recently the FDA released guidance on biopharmaceutical companies’ ability to share certain information about the potential benefits of a treatment with insurers and health systems. While that guidance was an important first step, to spur more value-based arrangements we also need to reform outdated, unclear regulations that can discourage companies from offering new types of discounts or taking on more risk in new payment arrangements.

PROTECTING AND SUPPORTING INNOVATION

In order to encourage continued innovation and accelerate the introduction of new medicines for patients, the FDA should continue driving efficiencies aimed at enhancing the prescription drug development process and ensuring timely regulatory review. Additionally, the United States must continue to protect medical innovations through a strong patent system while also ensuring international trading partners value and support biopharmaceutical innovation. Reducing the time and complexity for developing innovative medicines and protecting the intellectual property behind them will further support the new era of medicine benefiting patients’ lives.

STRENGTHENING THE HEALTH CARE SYSTEM

The market-based U.S. health care system has worked well over time, but more can be done to help the system work even better for patients. The 340B program needs significant reform as it is widely understood to distort the market and create incentives that raise health care costs. Modernizing the program is essential to ensure it is sustainable and benefits vulnerable or uninsured patients. Likewise, improving affordability and predictability for seniors in Medicare Part D could help strengthen the health care market and improve affordable access to medicines for patients.

COMBATING PRESCRIPTION DRUG ABUSE

The opioid crisis is too broad and complex for any one person or group to solve alone. There are many different factors driving the current public health crisis that must be addressed holistically, including preventing overprescribing, improving patient and prescriber education, expanding access to appropriate treatments and enhancing law enforcement capabilities to crack down on counterfeit fentanyl and other illegal drugs.

Profile Toolkit: Innovation

Medicines are Transforming the Trajectory of Disease

Medicines yield important progress against some of the most challenging diseases of our time. Today, new medicines target the underlying causes of disease in ways never seen before, and diseases previously regarded as deadly are now manageable and even curable. In this new era of medicine, breakthrough science and personalized therapies are transforming the way we treat patients with a broad range of chronic and rare conditions. Looking forward, continued advances in biopharmaceutical innovation will be critical in addressing unmet need, improving public health and solving future health care challenges.

Progress against Disease

Medicines play a central role in transforming the trajectory of many debilitating diseases, resulting in decreased death rates, improved health outcomes and better quality of life for patients.

• Cardiovascular Disease: Since 1980, the death rate declined by nearly 60 percent.

• HIV/AIDS: Since the introduction of antiretroviral therapies, the disease has become a chronic and manageable condition and death rates have declined 88 percent.

• Hepatitis C: A broad range of treatments with cure rates approaching 100 percent are available for patients with all forms of the disease.

• Cancer: The U.S. has witnessed a 26 percent decline in cancer death rates since peaking in the 1990s through new treatments.

Recent Approvals

In 2017, the U.S. FDA approved 56 new medicines. Examples of novel therapies that became available to patients in 2017 include:

• First cell and gene therapies: The first chimeric antigen receptor T-cell therapies were approved for two advanced, rare forms of blood cancer. The first directly administered gene therapy was also approved to treat a rare inherited form of blindness.

• First medicine for primary progressive multiple sclerosis (PPMS): Ocrelizumab was approved to in 2017 to treat the 15 percent of MS patients with this devastating form of MS.

• First treatment for sickle cell disease in 20 years: A medicine was approved to treat sickle cell disease, an inherited blood disorder that causes debilitating pain and organ damage.

The Future Has Never Been Brighter

There are about 7,000 medicines in clinical development globally with the potential to impact U.S. patients. And across the medicines in the pipeline, 74 percent have the potential to be first-in-class treatments.

• Cancer: Cell and gene therapies, antibody-drug conjugates, immune checkpoint modulators, metabolic immunotherapies and vaccines are all showing tremendous promise. Today, there are 1,120 medicines and vaccines currently in development for cancer.

• Heart disease and stroke: Cardiovascular disease is the leading cause of death in the United States, affecting 92.1 million American adults. There are currently 200 medicines in development for heart disease and stroke.

• Neurological disorders: These disorders affect a broad range of conditions affecting the brain and nervous system—for example, epilepsy, migraine headaches, MS, Parkinson’s disease and Alzheimer’s disease. There are more than 500 medicines in development for neurological disorders.

Profile Toolkit: Savings

Better Use of Medicines Can Improve Health Outcomes and Reduce the Use of Costly Medical Care

Medicines play a central role in making our health care system more sustainable. Use of medicines can help patients avoid other costlier services, such as emergency room visits, hospital stays, surgeries and long-term care. Yet, despite the many health and economic benefits medicines provide, significant gaps in the appropriate use of medicines remain.

The Economic Burden of Chronic Disease is Substantial

Medicines are enabling us to more effectively treat the biggest cost driver in health care: chronic disease. The cost of treating patients with chronic conditions accounts for 90 percent of the nearly $3 trillion spent on health care in the United States each year.

Significant Gaps in Optimal Use of Medicines

Nearly 75 percent of American adults do not follow their physicians’ prescription orders, including not filling their prescriptions or taking less than the recommended dose, and just 50 percent of medications for chronic disease are taken as prescribed.

But the growing use of high deductibles and coinsurance for medicines as well as other hurdles present affordability challenges for many patients. Such access restrictions to medicines can lead to patients not adhering to prescribed treatment regimens, resulting in poor outcomes.

Better Use of Medicines Can Improve Health Outcomes and Reduce Health Care Spending

Fortunately, where there are gaps there are also tremendous opportunities to drive value in our health care system. In fact, better use of medicines could eliminate $213 billion in U.S. health care costs annually, amounting to eight percent of the nation’s health care costs. For example, spending $1 more on medicines for adherent patients with congestive heart failure, high blood pressure, diabetes or high cholesterol can generate $3 to $10 in savings on emergency room visits and inpatient hospitalizations.

In addition to producing savings from avoided medical services, better use of medicines also improves health and overall quality of life, which can lead to improved productivity from lower disability and fewer missed days of work.

Today and in the future, medicines will play a central role in making our health care more sustainable. In consideration of the many diseases where there is significant unmet need, the development of medicines will continue to be critical in addressing the most costly and challenging diseases of our time.

Profile Toolkit: Economy

The Biopharmaceutical Industry’s Role in Fueling the Economy and Global Competitiveness

The U.S. is the global leader of biopharmaceutical innovation. Beyond the value medicines deliver to patients is the impact the biopharmaceutical sector has on local, state and national economies.

The economic impact of the biopharmaceutical industry translates into high-wage jobs, substantial tax revenue and growing economic output in our local communities. The combined effects of biopharmaceutical direct jobs, supply chain and wages and benefits resulted in $1.3 trillion in economic output and 4.7 million jobs in 2015. Every job in the biopharmaceutical industry supported nearly five additional jobs

America's biopharmaceutical Industry Is the Most Research Intensive

The biopharmaceutical industry is the global leader in R&D. It invests 12 times more in R&D per employee, employs the largest share of manufacturers and invests more in R&D relative to sales than most other manufacturing industries.

Biopharmaceutical companies based in the U.S. invested about $90 billion in R&D in 2016.

The pharmaceutical Industry is one of the most research-intensive industries in the United States. Pharmaceutical firms invest as much as five times more in research and development, relative to their sales, than the average manufacturing firm.”

Congressional Budget Office

The U.S. Biopharmaceutical Industry Is the Global Leader in Biomedical Innovation

America’s robust R&D enterprise is the envy of the world. The U.S. leads in overall clinical trial activity, early stage clinical research and more.

Like other R&D-intensive industries, the biopharmaceutical industry faces mounting competition not just from developed countries, but also from emerging nations. Looking ahead, we need to strengthen U.S. economic foundations and compete for biopharmaceutical development.

Since 2000, the FDA approved nearly 800 new medicines.

More than 3/4 of prescription drugs approved in 2017 were approved in U.S. before other countries.

There are about 7,000 medicines in development globally.

The United States is now facing increasing competition to attract and grow a biopharmaceutical presence not just from developed countries, but also from emerging nations such as Brazil, China, and Singapore that are laying the groundwork for future growth.”"

TEConomy Partners

Profile Toolkit: R&D

The Dynamic U.S. Research and Development Ecosystem

The cost, time and complexities of biopharmaceutical research have increased, introducing additional challenges in the research and development and manufacturing processes. As scientific complexities create new challenges, research-based biopharmaceutical companies are committed to realizing the promise of the pipeline and are working with stakeholders across the dynamic U.S. R&D ecosystem to leverage new scientific and technological advances to bring innovative medicines to patients. With about 7,000 medicines in clinical development globally – of which 74 percent have the potential to be first-in-class treatments – the future has never been brighter.

America's Biopharmaceutical Companies Play Central Role in R&D Ecosystem

U.S. biopharmaceutical companies play a central role in the biomedical research ecosystem. The complex system is marked by collaborations across industry, academic institutions, government agencies, venture capital firms, nonprofit foundations and others. This diverse group of stakeholders works together to advance science from the bench to the bedside. Early scientific advances form the foundation for the development of new medicines, which is led by biopharmaceutical companies.

The Complex Biopharmaceutical R&D Process

It takes about 10 to 15 years for a medicine from the start of the R&D process to approval. And only 12 percent of medicines entering clinical trials are ultimately approved by the FDA.

The average cost to develop a new medicine is $2.6 billion, including the cost of failures. And these costs have more than doubled over the past decade. Over this same period, rapid scientific and technical advances, alongside increasing regulatory burdens, are resulting in more complex clinical trials. Despite, these challenges PhRMA member companies themselves have invested more than half a trillion dollars in R&D since 2000.

BIOPHARMACEUTICAL RESEARCH AND DEVELOPMENT PROCESS

DISCOVERY

PRECLINICAL DEVELOPMENT

CLINICAL DEVELOPMENT

Before moving to studies in humans, the company submits an Investigational New Drug Application to the FDA.

FDA REVIEW/FDA APPROVAL

If the medicine passes clinical trials, the sponsor submits a New Drug Application or Biologics License Application.

ONGOING STUDY OF THE MEDICINE

Research continues throughout the life of a medicine to monitor safety in usage and explores other potential uses.

The Evolving R&D Process

Researchers are constantly seeking to refine the R&D process in response to new scientific and technological advances and evolving regulatory requirements.

The 21st Century Cures Act and the Prescription Drug User Fee Act (PDUFA) helps ensure the FDA has the tools needed to better manage the significant emerging science and innovation of today to meet the challenges of tomorrow. Increasing acceptance of innovative clinical trial designs, appropriate integration of the patient perspective and advancing the use of real-world evidence all hold the potential to speed the development and regulatory review process and drive market competition.

While new scientific advances bring greater promise and complexity, the process is inherently fraught with a high degree of scientific and regulatory uncertainty, and there are often research setbacks. Despite these challenges, researchers remain committed to conquering challenging diseases.

Providing Hope to Patients

It is critical that we have a policy and regulatory environment that promotes innovation to fulfill the promise that these scientific opportunities represent for patients. To continue to advance medical discovery, we need to ensure a well-functioning, science-based regulatory system that keeps pace with the latest advances and ensures the timely review, approval and introduction of new medicines. This will be critical not only in improving the lives of patients, but also in maintaining U.S. global leadership in biomedical innovation and sustaining and growing U.S. jobs. Finally, it is important that we have robust intellectual property (IP) rights and incentives to foster investment in R&D and commercialization of new technologies, including enforcement of IP protections in the United States and abroad.

Additional PhRMA Toolkits

10 THINGS YOU SHOULD KNOW

Discussions about costs are important. Many patients struggle to access the medicine they need and have important questions about their costs. America’s biopharmaceutical companies are committed to working with policymakers to create and advance solutions that further enhance the marketplace, lower costs for patients and promote continued medical innovation. Learn more about medicine and spending costs in one of our newest toolkit.

FOLLOW THE DOLLAR

Examining how money flows through the system and how that impacts what patients pay at the pharmacy can help consumers and policymakers find answers to their questions about affordability and access to medicines. The prices wholesalers, pharmacies, PBMs, insurers and patients pay for a medicine all vary and are shaped by negotiations in the supply chain. Follow the dollar and learn more about how the prices of medicines are shaped in this toolkit.

VALUE BASED-CONTRACTS

Payers and biopharmaceutical companies are exploring a range of new value-based contracts that tie reimbursement for medicines more closely to value for individual patients. These voluntary, private arrangements include performance-based contracts that link payment to demonstrated patient outcomes, varying payment based on how a medicine is used and other forms of risk sharing. This toolkit presents the challenges and opportunities of value-based contracts.